It’s a devastating diagnosis that about 1,000 babies in the United States receive every year: hypoplastic left heart syndrome (HLHS). The hearts of children born with this condition are underdeveloped and have too few muscle cells to pump blood effectively throughout the body. The poor blood flow causes neurocognitive delays and severe physical limitations. Without major surgical interventions, it’s fatal.

Today the University of Minnesota Medical School’s Jop van Berlo, M.D., Ph.D., is looking to change the trajectory of life for kids born with HLHS as he works to develop a gene-based treatment for it. A $300,000, three-year Hartwell Individual Biomedical Research Award from The Hartwell Foundation is backing this work.

Van Berlo’s team will explore the use of genes to prompt the scarce heart cells to divide and multiply, allowing the underdeveloped heart to continue growing after the child is born. Van Berlo says this approach, which could minimize the risks of surgery and improve long-term outcomes, will likely have broader applications in treating other congenital heart defects.

“The regeneration of cardiac cells is some of the most exciting science I have ever worked on,” says van Berlo, who is affliated with the U’s Stem Cell Institute and holds the Lois and Richard King Assistant Professorship in Medicine. “Through this research, I hope to unlock the pediatric heart’s ability to form new cells, so children with HLHS can be active, thrive, and avoid heart failure.”

To learn how you can support this work, contact Jonna Schnettler at 612-624-5588 or jschnett@umn.edu.